Department of Rheumatology Immunology and Allergy, The Children's Hospital, Zhejiang University School of Medicine, National Clinical Research Center for Child Health, Hangzhou, China.
Department of Rheumatology, Children's Hospital of Fudan University, National Children's Medical Center, Shanghai, China.
Mod Rheumatol. 2022 Oct 15;32(6):1114-1121. doi: 10.1093/mr/roab119.
To evaluate the safety and efficacy of tocilizumab (TCZ) on refractory macrophage activation syndrome (rMAS) associated with systemic juvenile idiopathic arthritis (sJIA-rMAS).
We retrospectively reviewed the charts of 14 patients diagnosed with sJIA-rMAS, who were treated with TCZ after failing conventional therapies at three hospital centres from January 2016 to December 2020. Demographic, clinical, and laboratory characteristics were recorded at the onset of MAS, before TCZ (pre-TCZ), and 14 days after TCZ (post-TCZ).
The clinical manifestation of sJIA-rMAS included fever (100%), skin rashes (35.7%), lymphadenomegaly (42.9%), hepatomegaly (57.1%), splenomegaly (7.1%), gastrointestinal symptoms (28.6%), arthritis (14.3%), myalgia (28.6%), and polyserositis (14.3%). After TCZ treatment, fever (100%, 14/14), gastrointestinal symptoms (100%, 4/4), and myalgia (100%, 4/4) were significantly improved after 1 week (P < 0.05). Skin rashes, lymphadenomegaly, and arthritis also improved in many patients, but these parameters did not reach statistical significance. In post-TCZ group, decreases in levels of C-reactive protein, erythrocyte sedimentation rate, and serum ferritin of sJIA-rMAS were observed compared with pre-TCZ (P < 0.05). No disease relapse or fatality was recorded during the follow-up (25 months, range 3-60 months).
TCZ is safe and effective for the treatment of sJIA-rMAS after failure of conventional therapies.
评估托珠单抗（TCZ）治疗全身型幼年特发性关节炎相关难治性巨噬细胞活化综合征（rMAS）的安全性和疗效。
我们回顾性分析了 2016 年 1 月至 2020 年 12 月期间，在三个医院中心，14 例接受常规治疗失败的全身型幼年特发性关节炎相关难治性巨噬细胞活化综合征患者使用 TCZ 治疗的病例。记录 MAS 发病时、TCZ 治疗前（pre-TCZ）和 TCZ 治疗后 14 天（post-TCZ）的人口统计学、临床和实验室特征。
全身型幼年特发性关节炎相关难治性巨噬细胞活化综合征的临床表现包括发热（100%，14/14）、皮疹（35.7%）、淋巴结肿大（42.9%）、肝肿大（57.1%）、脾肿大（7.1%）、胃肠道症状（28.6%）、关节炎（14.3%）、肌痛（28.6%）和多浆膜炎（14.3%）。TCZ 治疗后，发热（100%，14/14）、胃肠道症状（100%，4/4）和肌痛（100%，4/4）在 1 周内明显改善（P < 0.05）。许多患者的皮疹、淋巴结肿大和关节炎也有改善，但这些参数没有达到统计学意义。在 post-TCZ 组中，与 pre-TCZ 相比，sJIA-rMAS 的 C 反应蛋白、红细胞沉降率和血清铁蛋白水平均下降（P < 0.05）。在随访期间（25 个月，范围 3-60 个月），未记录到疾病复发或死亡。
在常规治疗失败后，TCZ 治疗全身型幼年特发性关节炎相关难治性巨噬细胞活化综合征是安全有效的。
